Cholesterol redistribution triggered by CYP46A1 gene therapy improves major hallmarks of Niemann-Pick type C disease but is not sufficient to halt neurodegeneration.
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| Title: | Cholesterol redistribution triggered by CYP46A1 gene therapy improves major hallmarks of Niemann-Pick type C disease but is not sufficient to halt neurodegeneration. |
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| Authors: | Nunes MJ; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal; Department of Pharmaceutical Sciences and Medicines, Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal., Carvalho AN; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal; Department of Pharmaceutical Sciences and Medicines, Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal., Reis J; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal., Costa D; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal., Moutinho M; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal., Mateus J; Instituto de Farmacologia e Neurociências, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal; Instituto de Medicina Molecular João Lobo Antunes, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal., Mendes de Almeida R; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal., Brito S; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal., Risso D; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal., Nunes S; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal., Castro-Caldas M; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal; Department of Life Sciences, Faculty of Science and Technology, Universidade NOVA de Lisboa, Caparica, Portugal., Gama MJ; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal; Department of Pharmaceutical Sciences and Medicines, Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal., Rodrigues CMP; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal; Department of Pharmaceutical Sciences and Medicines, Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal., Xapelli S; Instituto de Farmacologia e Neurociências, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal; Instituto de Medicina Molecular João Lobo Antunes, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal., Diógenes MJ; Instituto de Farmacologia e Neurociências, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal; Instituto de Medicina Molecular João Lobo Antunes, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal., Cartier N; NeuroGenCell, INSERM U1127, Paris Brain Institute (ICM), Sorbonne University, CNRS, APHP, University Hospital Pitié Salpêtrière, Paris, France., Chali F; NeuroGenCell, INSERM U1127, Paris Brain Institute (ICM), Sorbonne University, CNRS, APHP, University Hospital Pitié Salpêtrière, Paris, France., Piguet F; NeuroGenCell, INSERM U1127, Paris Brain Institute (ICM), Sorbonne University, CNRS, APHP, University Hospital Pitié Salpêtrière, Paris, France., Rodrigues E; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal; Department of Pharmaceutical Sciences and Medicines, Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal. Electronic address: Elsa.Rodrigues@ff.ulisboa.pt. |
| Source: | Biochimica et biophysica acta. Molecular basis of disease [Biochim Biophys Acta Mol Basis Dis] 2024 Mar; Vol. 1870 (3), pp. 166993. Date of Electronic Publication: 2023 Dec 22. |
| Publication Type: | Journal Article; Research Support, Non-U.S. Gov't |
| Journal Info: | Publisher: Elsevier Country of Publication: Netherlands NLM ID: 101731730 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1879-260X (Electronic) Linking ISSN: 09254439 NLM ISO Abbreviation: Biochim Biophys Acta Mol Basis Dis Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
| FullText | Text: Availability: 0 |
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| Header | DbId: mdl DbLabel: MEDLINE Ultimate An: 38142760 AccessLevel: 2 PubType: Academic Journal PubTypeId: academicJournal PreciseRelevancyScore: 0 |
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| Items | – Name: Title Label: Title Group: Ti Data: Cholesterol redistribution triggered by CYP46A1 gene therapy improves major hallmarks of Niemann-Pick type C disease but is not sufficient to halt neurodegeneration. – Name: Author Label: Authors Group: Au Data: <searchLink fieldCode="AU" term="%22Nunes+MJ%22">Nunes MJ</searchLink>; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal; Department of Pharmaceutical Sciences and Medicines, Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal.<br /><searchLink fieldCode="AU" term="%22Carvalho+AN%22">Carvalho AN</searchLink>; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal; Department of Pharmaceutical Sciences and Medicines, Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal.<br /><searchLink fieldCode="AU" term="%22Reis+J%22">Reis J</searchLink>; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal.<br /><searchLink fieldCode="AU" term="%22Costa+D%22">Costa D</searchLink>; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal.<br /><searchLink fieldCode="AU" term="%22Moutinho+M%22">Moutinho M</searchLink>; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal.<br /><searchLink fieldCode="AU" term="%22Mateus+J%22">Mateus J</searchLink>; Instituto de Farmacologia e Neurociências, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal; Instituto de Medicina Molecular João Lobo Antunes, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal.<br /><searchLink fieldCode="AU" term="%22Mendes+de+Almeida+R%22">Mendes de Almeida R</searchLink>; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal.<br /><searchLink fieldCode="AU" term="%22Brito+S%22">Brito S</searchLink>; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal.<br /><searchLink fieldCode="AU" term="%22Risso+D%22">Risso D</searchLink>; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal.<br /><searchLink fieldCode="AU" term="%22Nunes+S%22">Nunes S</searchLink>; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal.<br /><searchLink fieldCode="AU" term="%22Castro-Caldas+M%22">Castro-Caldas M</searchLink>; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal; Department of Life Sciences, Faculty of Science and Technology, Universidade NOVA de Lisboa, Caparica, Portugal.<br /><searchLink fieldCode="AU" term="%22Gama+MJ%22">Gama MJ</searchLink>; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal; Department of Pharmaceutical Sciences and Medicines, Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal.<br /><searchLink fieldCode="AU" term="%22Rodrigues+CMP%22">Rodrigues CMP</searchLink>; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal; Department of Pharmaceutical Sciences and Medicines, Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal.<br /><searchLink fieldCode="AU" term="%22Xapelli+S%22">Xapelli S</searchLink>; Instituto de Farmacologia e Neurociências, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal; Instituto de Medicina Molecular João Lobo Antunes, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal.<br /><searchLink fieldCode="AU" term="%22Diógenes+MJ%22">Diógenes MJ</searchLink>; Instituto de Farmacologia e Neurociências, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal; Instituto de Medicina Molecular João Lobo Antunes, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal.<br /><searchLink fieldCode="AU" term="%22Cartier+N%22">Cartier N</searchLink>; NeuroGenCell, INSERM U1127, Paris Brain Institute (ICM), Sorbonne University, CNRS, APHP, University Hospital Pitié Salpêtrière, Paris, France.<br /><searchLink fieldCode="AU" term="%22Chali+F%22">Chali F</searchLink>; NeuroGenCell, INSERM U1127, Paris Brain Institute (ICM), Sorbonne University, CNRS, APHP, University Hospital Pitié Salpêtrière, Paris, France.<br /><searchLink fieldCode="AU" term="%22Piguet+F%22">Piguet F</searchLink>; NeuroGenCell, INSERM U1127, Paris Brain Institute (ICM), Sorbonne University, CNRS, APHP, University Hospital Pitié Salpêtrière, Paris, France.<br /><searchLink fieldCode="AU" term="%22Rodrigues+E%22">Rodrigues E</searchLink>; Research Institute for Medicines (iMed.ULisboa), Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal; Department of Pharmaceutical Sciences and Medicines, Faculty of Pharmacy, Universidade de Lisboa, Lisbon, Portugal. Electronic address: Elsa.Rodrigues@ff.ulisboa.pt. – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%22101731730%22">Biochimica et biophysica acta. Molecular basis of disease</searchLink> [Biochim Biophys Acta Mol Basis Dis] 2024 Mar; Vol. 1870 (3), pp. 166993. <i>Date of Electronic Publication: </i>2023 Dec 22. – Name: TypePub Label: Publication Type Group: TypPub Data: Journal Article; Research Support, Non-U.S. Gov't – Name: TitleSource Label: Journal Info Group: Src Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Elsevier%22">Elsevier </searchLink><i>Country of Publication: </i>Netherlands <i>NLM ID: </i>101731730 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1879-260X (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2209254439%22">09254439 </searchLink><i>NLM ISO Abbreviation: </i>Biochim Biophys Acta Mol Basis Dis <i>Subsets: </i>MEDLINE |
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| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.1016/j.bbadis.2023.166993 Languages: – Code: eng Text: English PhysicalDescription: Pagination: StartPage: 166993 Titles: – TitleFull: Cholesterol redistribution triggered by CYP46A1 gene therapy improves major hallmarks of Niemann-Pick type C disease but is not sufficient to halt neurodegeneration. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Nunes MJ – PersonEntity: Name: NameFull: Carvalho AN – PersonEntity: Name: NameFull: Reis J – PersonEntity: Name: NameFull: Costa D – PersonEntity: Name: NameFull: Moutinho M – PersonEntity: Name: NameFull: Mateus J – PersonEntity: Name: NameFull: Mendes de Almeida R – PersonEntity: Name: NameFull: Brito S – PersonEntity: Name: NameFull: Risso D – PersonEntity: Name: NameFull: Nunes S – PersonEntity: Name: NameFull: Castro-Caldas M – PersonEntity: Name: NameFull: Gama MJ – PersonEntity: Name: NameFull: Rodrigues CMP – PersonEntity: Name: NameFull: Xapelli S – PersonEntity: Name: NameFull: Diógenes MJ – PersonEntity: Name: NameFull: Cartier N – PersonEntity: Name: NameFull: Chali F – PersonEntity: Name: NameFull: Piguet F – PersonEntity: Name: NameFull: Rodrigues E IsPartOfRelationships: – BibEntity: Dates: – D: 01 M: 03 Text: 2024 Mar Type: published Y: 2024 Identifiers: – Type: issn-electronic Value: 1879-260X Numbering: – Type: volume Value: 1870 – Type: issue Value: 3 Titles: – TitleFull: Biochimica et biophysica acta. Molecular basis of disease Type: main |
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